bluebird bio

Gene therapy’s potential could soon be fulfilled at last


Gene therapy has been promising to transform healthcare for half a century, and still we have only around ten treatments approved for use. But that is going to change dramatically over the next five to ten years, according to Nicola Redfern, UK general manager of bluebird bio, as many treatments for rare genetic diseases are brought forward.

In her introductory remarks for a digital round-table, hosted by The Spectator, Redfern laid out the challenges for the NHS and wider healthcare sector. ‘Have we got enough beds for transplant services?’ she says. ‘Can fertility services cope as patients may want eggs and sperm harvested?’

A major issue is the sheer cost of gene therapy. According to Professor Rafael J Yáñez-Muñoz, director of the Centre of Gene and Cell Therapy at Royal Holloway, University of London, the world’s most expensive medicine is a gene therapy treatment for spinal muscular atrophy, which weighs in at $2.1 million per patient in the US. And that’s just for the infusion of medicine — it excludes the business of delivering it to the patient. It is often a hugely complex process, putting considerable pressure on nursing teams, added Parker Moss of Genomics England.

Therapy administered to a child for a rare genetic disease might bring 50 or more years of benefit

That could still turn out to be good value, however. Many gene therapy treatments promise to be one-off affairs, or at worst may need to be delivered every five to ten years. Yet they could save a lifetime of medical costs. ‘If you took the one-off cost and divided it by the number of years benefit that patients may potentially get than the annual costs when you quantified it, it would be a lot less for gene therapies than it is for a lot of other medication and treatments we happily and regularly embrace,’ says Redfern.

Many gene therapies will be focused on the very young, for whom they could promise lifelong benefit. A good cancer drug might offer a patient 15 to 20 extra years of life, says Ross Selby, UK access value and evidence strategy lead for bluebird bio. By contrast, a gene therapy administered to a child for a rare genetic disease might bring 50 or more years of benefit.

The trouble is that Britain’s system of calculating the costs and benefits of novel drugs and treatments does not cope well with the concept of very expensive, one-off treatments. The National Institute for Health and Care Excellence (Nice), which judges whether treatments are sufficient value for money to be offered by the NHS, is set up more to assess the cost of repeat treatments (see p8). ‘Cell and gene therapies require a completely new economic model,’ says Parker Moss. We need to find ways of spreading costs over the years, he added, so that the NHS pays in instalments, hopefully out of money it is saving by not having to provide continuous treatment for a cured patient. We need, too, a payment model where the NHS and the pharmaceutical sector can share the efficacy risks.

To prove the efficacy of a treatment which is sold as a lifelong cure for a serious genetic condition is not easy. There is a danger of overselling gene therapy as a ‘silver bullet’ for all manner of conditions. ‘The hope it offers is exceeding the reality of what’s available right now,’ says Emily Crossley, CEO of Duchenne UK. Nevertheless, she would regard it as a ‘cure’ if her own son, who suffers from Duchenne muscular dystrophy, could live until 70 and walk until his late thirties. Currently, his life expectancy is his late twenties.

Healthcare bodies must be realistic and not drag their heels. It took Nice two and a half years to approve a treatment for Duchenne. During that delay, says Crossley, boys became ineligible for the medicine, which was heartbreaking for the families hoping to give their sons the drug and seeing countries in Europe paying for it.

Yáñez-Muñoz, though, has cautionary advice for pharmaceutical companies bringing gene therapies to the market. ‘We need companies to be more open about how the price of these drugs is worked out,’ he says. ‘We need to find a way so that it is profitable for the companies, but also ethical.’ This is not least, he says, because most gene therapies have been initially developed by academic labs funded by governments and charities before being promoted by pharma, and tested in public hospitals on volunteers in trials. While clinical trials are subject to ethical approval, independent review and stringent regulatory standards of safety, these volunteers still make sacrifices to take part and know that they are probably not the beneficiary in the longer term.

Some 12 per cent of trials globally are being conducted in Britain, according to Matthew Durdy, CEO of the government’s Cell and Gene Therapy Catapult. The availability of money to support trials, and a Stevenage-based manufacturing centre for drugs used in those trials, set up by the Cell and Gene Therapy Catapult, is continuing to attract therapy developers to what is currently the largest cell and gene therapy cluster outside the US.

For years, says Professor Antonio Pagliuca of King’s College Hospital NHS Trust, cell and gene therapy in Britain was ‘an academic spectator sport’. ‘Individuals were beavering away with support from grant-awarding bodies with many not seeing the potential of it.’ Now, however, says Ian Campbell, interim executive chair of Innovate UK, we have a good research infrastructure. Clinical trials which used to take two years to win approval can now get clearance in weeks or months.

The big question is: can Britain stay a leader in the field? ‘The UK hits well above its weight,’ says Pagliuca. ‘Now we’ve got to avoid doing what we commonly do, which is to innovate, show the world — and then take a back seat while someone commercialises it.’

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Hosted digitally on Monday 26th October, this roundtable was sponsored by bluebird bio and featured the following guests; Kate Andrews, Economics Correspondent, The Spectator (chair); Emily Crossley, CEO, Duchenne UK; Matthew Durdy, CEO, Cell and Gene Therapy Catapult; Dr Ian Campbell, Interim Executive Chair, Innovate UK; Parker Moss, Genomics England; Professor Antonio Pagliuca, King’s College Hospital NHS Foundation Trust; Nicola Redfern, UK General Manager, bluebird bio; Ross Selby, UK Access Value and Evidence Strategy Lead, bluebird bio; David Taylor, Professor Emeritus of Pharmaceutical and Public Health Policy; Rachel Wolf, Founding Partner, Public First; Lawrence Woolard, Founder and Director, On The Pulse; Professor and Rafael J Yáñez-Muñoz, Director of Centre of Gene and Cell Therapy, Royal Holloway University.

This article first appeared in a Spectator supplement: The evolution of gene therapy. Sponsored by bluebird bio.


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